Human stem cells harboring a suicide gene improve the safety and standardisation of neural transplants in Parkinsonian rats.

ABSTRACT: Despite advancements in human pluripotent stem cells (hPSCs) differentiation protocols to generate appropriate neuronal progenitors suitable for transplantation in Parkinson's disease, resultant grafts contain low proportions of dopamine neurons. Added to this is the tumorigenic risk associated with the potential presence of incompletely patterned, proliferative cells within grafts. Here, we utilised a hPSC line carrying a FailSafe^TM suicide gene (thymidine kinase linked to cyclinD1) to selectively ablate proliferative cells in order to improve safety and purity of neural transplantation in a Parkinsonian model. The engineered FailSafe^TM hPSCs demonstrated robust ventral midbrain specification in vitro, capable of forming neural grafts upon transplantation. Activation

SUBMITTER: de Luzy IR 

PROVIDER: S-EPMC8160354 | biostudies-literature | 2021 May

REPOSITORIES: biostudies-literature